ERDERA has received funding from the European Union’s Horizon Europe research and innovation programme under grant agreement N°101156595.
Views and opinions expressed are those of the author(s) only and do not necessarily reflect those of the European Union or any other granting authority, who cannot be held responsible for them.
© 2025 | ERDERA — European Rare Diseases Research Alliance
ERDERA is coordinated by Insem
The RE(ACT) Congress & IRDiRC Conference 2025, an international platform for knowledge sharing on rare and orphan diseases, has been a catalyst for scientific collaboration and advancement since its inception in 2012.
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) have jointly issued a clear warning about unregulated Advanced Therapy Medicinal Products (ATMPs). While these therapies—based on genes, tissues or cells—offer genuine promise, its limited regulation procedures raise serious health and safety concerns. Authorities urge patients to confirm that any ATMP is approved through official channels to avoid potential risks.
We met Irene Norstedt, Director for the People Directorate at DG Research and Innovation in the European Commission, at the RE(ACT) Congress in Brussels, where she delivered a compelling presentation on diverse funding models targeting different research needs.
The European Medicines Agency (EMA), together with the European Medicines Regulatory Network, has issued a renewed call for registrations in their Real-World Data (RWD) Catalogues, marking one year since the initiative’s launch. This publicly accessible online resource replaced the previous ENCePP Resources Database and the EU PAS Register, consolidating metadata on RWD sources and studies to aid regulators, researchers, and pharmaceutical companies in assessing the real-world use, safety, and effectiveness of medicines. EMA aims to build a comprehensive resource by encouraging stakeholders who manage data sources or who have conducted RWD studies to register their metadata. Participation not only increases READ MORE
The 8th RE(ACT) Congress and the 6th IRDiRC (International Rare Diseases Research Consortium) Conference concluded today, marking the end of a three-day summit where scientists, patient advocates, funders, and policymakers tackled the rare disease field’s most pressing challenges.
On 28 February, coinciding with the 2025 Rare Disease Day, Parliament’s public health committee, SANT, launched an online survey to gather insights on rare diseases. The initiative aims to build a detailed understanding of the challenges faced by individuals affected by rare diseases, as well as to collect views from stakeholders—including patients, caregivers, industry representatives, READ MORE
Imagine having a disease so rare that doctors struggle to diagnose it, researchers have little data to study it, and treatments are scarce. For the 30 million people in Europe living with a rare disease, this is a daily reality. But change is happening. READ MORE
The landscape of clinical research and genomic data governance is evolving rapidly, with two significant developments shaping the future of global health research and regulatory frameworks: the ongoing revision of the ICH E6 (R3) Good Clinical Practice (GCP) guideline and the recent release of WHO guidance on the ethical collection, access, use, and sharing of READ MORE
We are pleased to announce that the platform for submitting project applications for the Joint Transnational Call for Proposals (JTC) 2025 is now officially live. This highly anticipated call invites research teams across Europe and internationally to collaborate on cutting-edge projects aimed at “Pre-clinical therapy studies for rare diseases using small molecules and biologicals – READ MORE
For decades, rare disease patients and their families have endured a painful “diagnostic odyssey,” bouncing between specialists and often going years without answers. Rare diseases, by definition, affect fewer than 5 in 10,000 people in the European Union, but with over 7,000 such conditions cataloged, their collective impact is profound. A staggering 70% of these READ MORE
We are delighted to announce that the Ministry of Health of the Republic of Slovenia (Ministrstvo za zdravje) has officially joined the funding organisations participating in the first Joint Transnational Call for Proposals on Rare Disease Therapies. This initiative is part of ERDERA’s ongoing commitment to advancing cutting-edge research in this critical area. READ MORE
Following the success of our previous session, ERDERA warmly invites researchers, patient advocates, and early-career professionals to an extended webinar on 14 January 2025. This webinar is part of the 2025 Joint Transnational Call for Proposals, “Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation”, which was officially launched on READ MORE
The European Rare Diseases Research Alliance (ERDERA) is delighted to announce the official launch of its 2025 Joint Transnational Call (JTC) for Proposals as of today, 10 December 2024. This eagerly anticipated call invites research teams across Europe and internationally to collaborate on cutting-edge projects aimed at “Pre-clinical therapy studies for rare diseases using small READ MORE
